Type 1 Diabetes (T1D) Clinical Trials
The Faustman Lab is conducting clinical trials in long-term type 1 diabetes through the BCG Human Clinical Trial Program. This program is testing Bacillus Calmette-Guérin (BCG), an inexpensive generic drug that temporarily elevates levels of TNF (a signaling protein involved in the body’s immune responses), to see if it will benefit patients living with type 1 diabetes by eliminating the disease-causing T cells that attack and destroy the insulin-secreting cells of the pancreas.
Under the direction of Dr. Faustman and David Nathan, MD, director of the MGH Diabetes Center, a double-blind, placebo-controlled Phase I human clinical trial was conducted and demonstrated that BCG vaccination is not only safe in individuals with advanced type 1 diabetes, but may also be effective in reversing long-term disease. In the study, BCG was administered to adults who had been living with type 1 diabetes for an average of 15 years. Treatment helped eliminate the defective T cells that mistakenly attack and destroy the insulin-producing cells of the pancreas, also temporarily restoring the ability of the pancreas to produce small amounts of insulin. The results of the Phase I study were published in 2012.
Major Findings from the Phase I Trial
The major findings from the Phase I study were:
• The BCG vaccine with multi-dosing was safe in advanced type 1 diabetes.
• Although the drug was given in relatively small doses, we saw targeted death of the “bad” T cells that attack the insulin-secreting islets, an early sign that BCG has the potential to stop the autoimmune attack and successfully reverse disease.
• In people living with diabetes for an average of 15 years, there was a transient increase in/restoration of pancreatic insulin secretion after BCG vaccination.
A Phase II study is currently being planned. The goal of the Phase II trial will be to identify the dose and schedule of BCG vaccination that will put advanced type 1 diabetes into remission and prevent long-term diabetic complications. For long-term restoration of insulin secretion, we expect that more frequent or higher BCG dosing will be needed compared to that used in the Phase I study.